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Related Concept Videos

Pharmacogenomics: Identification of New Drug Targets01:29

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Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
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Genetic screens are tools used to identify genes and mutations responsible for phenotypes of interest. Genetic screens help identify individuals or a group of people at risk of developing  genetic diseases and help them with early intervention, targeted therapy, and reproductive options.
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To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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Related Experiment Video

Updated: Feb 19, 2026

Author Spotlight: Finding New Therapeutic Targets for Malignant Peripheral Nerve Sheath Tumor Through Genome-Scale shRNA Screens
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Target Discovery for Precision Medicine Using High-Throughput Genome Engineering.

Xinyi Guo1,2, Poonam Chitale1,2, Neville E Sanjana3,4

  • 1New York Genome Center, 101 Avenue of the Americas, New York, NY, 10013, USA.

Advances in Experimental Medicine and Biology
|November 14, 2017
PubMed
Summary
This summary is machine-generated.

High-throughput CRISPR screens enable precise genome editing for discovering therapeutic targets. These advanced CRISPR/Cas9 tools identify genes involved in cancer, infectious diseases, and genetic disorders.

Keywords:
CancerDrug resistanceFunctional genomicsGenome engineeringInfectious diseaseMetabolismPooled CRISPR screensTarget identification

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Area of Science:

  • Genomics
  • Molecular Biology
  • Biotechnology

Background:

  • Programmable RNA-guided nucleases, like CRISPR/Cas9, have revolutionized precision genome editing.
  • Functional screens utilize large libraries of RNA guides to explore biological processes and disease phenotypes.

Purpose of the Study:

  • To review recent advancements in high-throughput CRISPR screens.
  • To highlight the potential of these screens in identifying novel therapeutic targets.

Main Methods:

  • Utilizing CRISPR/Cas9 for genome-wide functional screens.
  • Employing large libraries of RNA guides for loss-of-function and gain-of-function screens.
  • Targeting noncoding genetic elements to understand their roles.

Main Results:

  • CRISPR screens can pinpoint genes and genetic elements critical to biological functions.
  • These screens are effective in interrogating complex biological questions and disease mechanisms.
  • Recent screens have demonstrated success in identifying targets for various diseases.

Conclusions:

  • High-throughput CRISPR screens offer powerful tools for biological discovery.
  • These screens have significant potential for uncovering novel therapeutic targets in areas like cancer and infectious diseases.
  • CRISPR technology is advancing the identification of targets for genetic disorders and other therapeutic challenges.